Implications of haemophilia gene therapy for the changing role of the multidisciplinary team

Miesbach, Wolfgang;Barcenilla, Sara Garcia;Golan, Gaby;Lobet, Sébastien
(2022) Haemophilia (Print) — Vol. 28, n° 1, p. e12-e14 (2022)

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Authors
  • Miesbach, Wolfgang
    Author
  • Barcenilla, Sara Garcia
    Author
  • Golan, Gaby
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  • Lobet, SébastienUCLouvain
    Author
Abstract
(en) Gene therapies (GT) for haemophilia are in clinical trials, and under review by regulatory agencies with an expectation they may become available in clinical practice over the coming years. This new treatment modality represents a paradigm shift in care for people with haemophilia (PwH), and will have an impact on the traditional set up of haemophilia treatment centres (HTCs). A recent joint statement from EAHAD and EHC recommended that first‐generation GT should be managed using a hub‐and‐spoke model, with GT prescribed and managed exclusively in expert comprehensive care centres (the hub), and monitored in HTCs (the spokes) in close communication. [...]
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Miesbach, W., Barcenilla, S. G., Golan, G., & Lobet, S. (2022). Implications of haemophilia gene therapy for the changing role of the multidisciplinary team. Haemophilia (Print), 28(1), e12-e14. https://doi.org/10.1111/hae.14440 (Original work published 2022)